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lüll Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment Pai SY; Notarangelo LDImmunol Allergy Clin North Am 2010[May]; 30 (2): 179-94The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder characterized by a triad of diagnostic clinical elements: immunodeficiency, eczema, and hemorrhage caused by thrombocytopenia with small-sized platelets. The formal proof that hematopoietic cell transplantation (HCT) could be used to cure WAS revealed a requirement for both immunosuppression and myelosuppression that still underlies the standard approach to curative therapy today. The current short- and long-term toxicities of HCT are the main stumbling block for the ability to cure every patient with WAS and X-linked thrombocytopenia, and much remains to be done.|*Hematopoietic Stem Cell Transplantation[MESH]|Animals[MESH]|Antibodies, Monoclonal, Murine-Derived[MESH]|Antibodies, Monoclonal/therapeutic use[MESH]|CD40 Antigens/immunology[MESH]|Chemotherapy, Adjuvant[MESH]|Histocompatibility[MESH]|Humans[MESH]|Immunity, Cellular/drug effects[MESH]|Myeloablative Agonists/*therapeutic use[MESH]|Rituximab[MESH]|Stem Cell Niche[MESH]|Wiskott-Aldrich Syndrome/genetics/*immunology/*therapy[MESH] |